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Raptor Pharmaceutical: FDA Approval Of Procysbi

May 1, 2013

The FDA announces on April 30, 2013, the approval of Raptor Pharmaceutical’s orphan drug Procysbi (Cysteamine Bitartrate) for the treatment of Nephropathic Cystinosis in children and adults.

In December 2012, Procysbi’s FDA PDUFA date is extended by 3 months from January 30, 2013 to April 30, 2013. The FDA extension is to provide more time for the FDA to complete the review of information submitted by the company within 3 months of the original PDUFA date of January 30, 2013. No additional studies are requested.

Currently, Mylan’s orphan drug Cystagon (Cysteamine), approved in 1994 by the FDA, is the only FDA-approved treatment for Nephropathic Cystinosis. Both drugs are two different forms of Cysteamine Bitartrate. Cystagon is released immediately and must be taken on a strict schedule every six hours or 4 times a day. Procysbi is an oral delayed, extended-release form of Cysteamine Bitartrate that can be taken twice daily.

Raptor Pharmaceutical plans to launch Procysbi by the end of the 2nd Q 2013. The FDA approval of the drug triggers a $25 million payment to the company under a loan agreement with HealthCare Royalty Partners. Per a Motley Fool article, analysts see annual sales for Procysbi of about $150 million.

The Cystinosis Research Foundation (CFR) announces  on April 29, 2013, that it raises $2.2 million at its 11th anniversary celebration of Natalie’s Wish for research for the development of new treatments for Cystinosis. Per the CRF Press Release, “CRF funded every bench and clinical research study that led to the discovery of delayed-release cysteamine which was then reformulated by Raptor Pharmaceutical. The new formulation will cut the medication’s dosing frequency in half, to every 12 hours verses the current six-hour dosing schedule.”

Facts About Nephropathic Cystinosis

1) Inherited lysosomal storage disease causing “systemic toxic cystine accumulation which leads to progressive and irreversible tissue damage and multi-organ failure”

2) Usually diagnosed in infancy; usually kidney transplant by age 10 if untreated

3) Mylan Inc’s orphan drug Cystagon (Cysteamine) is the current Standard of Care (SOC)

4) Approximate WorldWide (WW) patient population of 2,000 – including 500 in US.


Cystinosis Foundation

Cystinosis Research Foundation (Natalie’s Wish)

Cystinosis Research Network (CRN) 

Raptor Pharmaceutical April 30, 2013 Press Release.

Please Note: FDA Official Logo from FDA website.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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